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Use of Pentamidine in Myotonic Dystrophy Models

Myotonic Dystrophy is characterized by 200–2000 CUG repeats in the 3’UTR of the DMPK gene—unaffected individuals have 5–35 CUG repeats. The CUG repeats form a stem-loop and it’s been suggested that this is a gain-of-function mutation wherein the stem loop binds proteins in the cell so that the proteins are unavailable to perform their normal cellular...

Published: 7/27/2009 | Updated: 5/22/2009 | Inventor(s): Andy Berglund, Bryan Warf, Leslie Vanos, Catherine Matthys

Keywords(s): Chemical, Therapeutics

Category(s): Research tools